Psychosine, a marker of Krabbe phenotype and treatment effect von Escolar, M.L., Kiely, B.T., Shawgo, E., Hong, X., Gelb, M.H., Orsini, J.J., Matern, D., Poe, M.D.

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Multidisciplinary Management of Hunter Syndrome von Muenzer, Joseph, Beck, M, Eng, C. M, Escolar, M. L, Giugliani, R, Guffon, N. H, Harmatz, P, Kamin, W, Kampmann, C, Koseoglu, S. T, Link, B, Martin, R. A, Molter, D. W, Munoz Rojas, M. V, Ogilvie, J. W, Parini, R, Ramaswami, U, Scarpa, M, Schwartz, I. V, Wood, R. E, Wraith, E

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Allogeneic hematopoietic SCT for alpha-mannosidosis: an analysis of 17 patients von Mynarek, M, Tolar, J, Albert, M H, Escolar, M L, Boelens, J J, Cowan, M J, Finnegan, N, Glomstein, A, Jacobsohn, D A, Kühl, J S, Yabe, H, Kurtzberg, J, Malm, D, Orchard, P J, Klein, C, Lücke, T, Sykora, K-W

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Diffusion Tensor Imaging Detects Abnormalities in the Corticospinal Tracts of Neonates with Infantile Krabbe Disease von Escolar, M.L, Poe, M.D, Smith, J.K, Gilmore, J.H, Kurtzberg, J, Lin, W, Styner, M

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Transplantation of Umbilical-Cord Blood in Babies with Infantile Krabbe's Disease von Escolar, Maria L, Poe, Michele D, Provenzale, James M, Richards, Karen C, Allison, June, Wood, Susan, Wenger, David A, Pietryga, Daniel, Wall, Donna, Champagne, Martin, Morse, Richard, Krivit, William, Kurtzberg, Joanne

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Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome von Staba, Susan L, Escolar, Maria L, Poe, Michele, Kim, Young, Martin, Paul L, Szabolcs, Paul, Allison-Thacker, June, Wood, Susan, Wenger, David A, Rubinstein, Pablo, Hopwood, John J, Krivit, William, Kurtzberg, Joanne

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Regional differences in fiber tractography predict neurodevelopmental outcomes in neonates with infantile Krabbe disease von Gupta, A, Poe, M D, Styner, M A, Panigrahy, A, Escolar, M L

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Early Clinical Markers of Central Nervous System Involvement in Mucopolysaccharidosis Type II von Holt, Joshua, BS, Poe, Michele D., PhD, Escolar, Maria L., MD

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Gross Motor Development of Children With Hurler Syndrome After Umbilical Cord Blood Transplantation von Dusing, Stacey C, Thorpe, Deborah E, Poe, Michele D, Rosenberg, Angela E, Mercer, Vicki S, Escolar, Maria L

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Temporal and Spatial Gait Characteristics of Children With Hurler Syndrome After Umbilical Cord Blood Transplantation von Dusing, Stacey C, Thorpe, Deborah E, Mercer, Vicki S, Rosenberg, Angela E, Poe, Michele D, Escolar, Maria L

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Excellent Outcomes Using Reduced-Intensity Conditioning for Patients with Inborn Errors of Immunity, Hematopoiesis, and Metabolism von Lugt, Mark Vander, MD, Chen, Xiaohua, MD, PhD, Chong, Hey Jin, MD, PhD, Windreich, Randy M., MD, Goyal, Rakesh K., MD, Escolar, Maria L., MD, Szabolcs, Paul, MD

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Forced Deflation PFT: A Novel Method to Evaluate Lung Function in Infants and Young Children von Goyal, Rakesh K., MD, Ibrahimova, Azada, MD, Escolar, Maria L., MD, Szabolcs, Paul, MD, Lugt, Mark Vander, MD, Windreich, Randy M., MD, Weiner, Daniel J., MD

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Randomized, blinded trial of weekend vs daily prednisone in Duchenne muscular dystrophy von ESCOLAR, D. M, HACHE, L. P, PESTRONK, A, RYAN, M. M, MONASTERIO, E, DAY, J. W, ZIMMERMAN, A, ARRIETA, A, HENRICSON, E, MAYHEW, J, FLORENCE, J, HU, F, CLEMENS, P. R, CONNOLLY, A. M, CNAAN, A, MCDONALD, C. M, VISWANATHAN, V, KORNBERG, A. J, BERTORINI, T. E, NEVO, Y, LOTZE, T

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Newborn screening for Krabbe disease in New York State: the first eight years’ experience von Orsini, Joseph J., Kay, Denise M., Saavedra-Matiz, Carlos A., Wenger, David A., Duffner, Patricia K., Erbe, Richard W., Biski, Chad, Martin, Monica, Krein, Lea M., Nichols, Matthew, Kurtzberg, Joanne, Escolar, Maria L., Adams, Darius J., Arnold, Georgianne L., Iglesias, Alejandro, Galvin-Parton, Patricia, Kronn, David F., Kwon, Jennifer M., Levy, Paul A., Pellegrino, Joan E., Shur, Natasha, Wasserstein, Melissa P., Caggana, Michele

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Cobomarsen, an Oligonucleotide Inhibitor of miR-155, Slows DLBCL Tumor Cell Growth In Vitro and In Vivo von Anastasiadou, Eleni, Seto, Anita G., Beatty, Xuan, Hermreck, Melanie, Gilles, Maud-Emmanuelle, Stroopinsky, Dina, Pinter-Brown, Lauren C., Pestano, Linda, Marchese, Cinzia, Avigan, David, Trivedi, Pankaj, Escolar, Diana M., Jackson, Aimee L., Slack, Frank J.

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A Randomized Study of Alglucosidase Alfa in Late-Onset Pompe's Disease von van der Ploeg, Ans T, Clemens, Paula R, Corzo, Deyanira, Escolar, Diana M, Florence, Julaine, Groeneveld, Geert Jan, Herson, Serge, Kishnani, Priya S, Laforet, Pascal, Lake, Stephen L, Lange, Dale J, Leshner, Robert T, Mayhew, Jill E, Morgan, Claire, Nozaki, Kenkichi, Park, Dorothy J, Pestronk, Alan, Rosenbloom, Barry, Skrinar, Alison, van Capelle, Carine I, van der Beek, Nadine A, Wasserstein, Melissa, Zivkovic, Sasa A

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Allogeneic stem cell transplantation corrects biochemical derangements in MNGIE von HIRANO, M, MARTI, R, SCHWARTZ, J, HAWKS, R. G, MARTONE, D. L, CAIRO, M. S, DIMAURO, S, STANZANI, M, GARVIN, J. H, SAVAGE, D. G, CASALI, C, TADESSE, S, ULDRICK, T, FINE, B, ESCOLAR, D. M, VALENTINE, M. L, NISHINO, I, HESDORFFER, C

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Macrophages Expressing GALC Improve Peripheral Krabbe Disease by a Mechanism Independent of Cross-Correction von Weinstock, Nadav I., Shin, Daesung, Dhimal, Narayan, Hong, Xinying, Irons, Eric E., Silvestri, Nicholas J., Reed, Chelsey B., Nguyen, Duc, Sampson, Oliver, Cheng, Yung-Chih, Lau, Joseph T.Y., Bongarzone, Ernesto R., Kofler, Julia, Escolar, Maria L., Gelb, Michael H., Wrabetz, Lawrence, Feltri, M. Laura

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Characterizing effective teaching in nursing pharmacology YouTube videos: A mixed methods study von Margallo, Maria Catalina M., Mariano, Carmela Nicole P., Martinez, Kryssia Nadine Fauve L., Mariano, Rashel Lidia T., Maramag, Lyra Mikhaela L., Mariano, Aira Angelica C., Mapue, Robel Christian L., Maturan, Earl Rae C., Escolar Chua, Rowena L.

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OP15.09: Circulating placenta‐derived extracellular vesicles contribute to endothelial dysfunction in pre‐eclampsia von Youssef, L., Ramos, A., Molina, P., Martinez‐Sanchez, J., Moreno‐Castaño, A., Blasco, M., De Moner, B., Tortajada, M., Camacho, M., Borrell, M., Crovetto, F., Pino, M., Escolar, G., Carreras, E., Gratacos, E., Diaz‐Ricart, M., Palomo, M., Crispi, F.

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